Baxter International Inc. (NYSE:BAX) today announced it has completed enrollment in its Phase III clinical trial of BAX 855, an investigational extended half-life, recombinant factor VIII (rFVIII) treatment for hemophilia A. The ongoing trial is aimed at assessing the efficacy of the compound in reducing annualized bleed rates (ABR) in both prophylaxis and on-demand treatment schedules, and will also evaluate its safety and pharmacokinetic profile.
BAX 855 was designed based on the full-length ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] molecule, a product with 10 years of real-world experience. The BAX 855 molecule was modified with PEGylation technology designed to extend its duration of activity in the body.
''The BAX 855 development program is a priority for Baxter as we evaluate the potential to provide an efficacious and safe treatment with an extended half-life for patients with hemophilia,'' said Anders Ullman, M.D., Ph.D., vice president of global research and development in Baxter’s BioScience business. ''We are focused first and foremost on strategies to address optimal efficacy and minimize patients’ bleeding episodes, while at the same time delivering on the convenience of less frequent dosing for this population with severe disease.''
The Phase II/III multi-center, open-label study called PROLONG-ATE is evaluating BAX 855 among 146 adult patients with previously-treated severe hemophilia A. Patients participating in PROLONG-ATE receive treatment twice weekly (45 IU/kg) and are followed for six months. The primary endpoint of the study is the annualized bleed rate (ABR) during the treatment period. The study is also evaluating the safety and immunogenicity of the compound when administered on either prophylaxis and on-demand treatment regimens. Other outcome measures include number of infusions needed to treat bleeding episodes, time intervals between these episodes, pharmacokinetics and patient reported outcomes. To date, no inhibitors or safety issues have been reported in the study.
Based upon the results of the study, the company expects to complete the trial and file for regulatory approval late in 2014. Baxter is also initiating a continuation study for all patients who complete the pivotal Phase II/III study, and expects to initiate a study of BAX 855 among pediatric patients in 2014.